Phase I trial

Arcellx has revealed encouraging interim data from its Phase I study of CART-ddBCMA in patients with relapsed or refractory multiple myeloma. The company plans to present new data with a median follow-up of 26.5 months at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition taking place 9-12 December 2023 in San Diego, […]

Asklepios BioPharmaceutical (AskBio), a gene therapy subsidiary of Bayer, has presented first-in-human data from a Phase I trial investigating AB-1002 (also known as NAN-101) for the treatment of patients with congestive heart failure (CHF). The data were shared at the American Heart Association Scientific Sessions, which were held in Philadelphia, US, 11-13 November 2023. The […]

Topline results from a Phase I/II clinical trial have shown that Kiora’s light-restoring small molecule KIO-301 has the potential to meaningfully improve vision in patients with retinitis pigmentosa (RP). The results of the ABACUS study for Kiora’s intravitreal (IVT) molecular photoswitch were presented at the American Academy of Ophthalmology annual conference by Russell Van Gelder, […]

Gate Neurosciences has announced positive topline quantitative EEG (qEEG) biomarker and safety results from its Phase I multiple ascending dose study of apimostinel in healthy volunteers. The results demonstrated a dose-dependent increase in qEEG pharmacodynamic biomarkers of NMDA receptor target activation from baseline, compared with subjects who received placebo. Maximal qEEG effects were observed at […]

Initial data from an ongoing Phase I/II study of HPK1 inhibitor NDI-101150 show clinical benefit in three patients with solid tumours, including one complete response in a patient with renal cell carcinoma. Two patients with pancreatic cancer and renal cell carcinoma, respectively, exhibited prolonged (> six months) stable disease. NDI-101150 demonstrated an acceptable safety profile […]

UK regulatory authorities have approved the first trial of a gene therapy for young children with Hunter syndrome. The UK Medicines and Healthcare Products Regulatory Agency (MHRA), Research Ethics Committee (REC), and Health Research Authority (HRA) have all approved the clinical trial application that was submitted by The University of Manchester in August 2022. In […]

The latest episode of the DDW Highlights podcast is now available to listen to below. DDW’s Megan Thomas narrates five key stories of the week to keep DDW subscribers up-to-date on the latest industry updates.  The news highlights this week feature a variety of treatment approaches, from cell therapy and antibody-drug conjugate to autotaxin inhibitor and arenaviral vaccine, […]

Kura Oncology’s FIT-001 Phase I dose-escalation trial of KO-2806, a farnesyl transferase inhibitor (FTI) for the treatment of advanced solid tumours, has commenced with the dosing of the first patient. FIT-001 is a first-in-human, multicentre, open-label study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of KO-2806 when administered as monotherapy and […]

News round-up for 23-27 October by DDW Digital Content Editor Diana Spencer. My news highlights this week feature a variety of treatment approaches, from cell therapy and antibody-drug conjugate to autotaxin inhibitor and arenaviral vaccine, all entering early in-human clinical trials to treat a range of diseases. The top stories: Natural killer cell therapy shows […]

Iksuda Therapeutics has dosed the first patient in a Phase I trial of antibody drug conjugate (ADC) IKS014 in patients with advanced solid tumours that express HER2. This follows positive data from Phase I clinical trials through Fosun Pharma, which is now progressing FS-1502 (IKS014) through Phase II and Phase III trials in China. The […]

Gamida Cell has revealed early data in 10 patients with CD20 positive non-Hodgkin lymphoma as part of an ongoing study of natural killer (NK) cell therapy candidate GDA-201. The multicentre Phase I study is designed to evaluate safety and determine the maximum tolerated dose. Enrolled patients were heavily pretreated with a median of six prior […]

The latest episode of the DDW Highlights podcast is now available to listen to below. DDW’s Megan Thomas narrates five key stories of the week to keep DDW subscribers up-to-date on the latest industry updates.  The buzz with week has been around Phase I trials, with new therapies for lung cancer, geographic atrophy, Parkinson’s, sarcopenia and long Covid […]

News round-up for 9-13 October by DDW Digital Content Editor Diana Spencer. The buzz with week has been around Phase I trials, with new therapies for lung cancer, geographic atrophy, Parkinson’s, sarcopenia and long Covid demonstrating positive early results or entering clinical trials for the first time. The top stories: Non-viral gene therapy shows early […]

Gain Therapeutics has initiated a Phase I clinical trial of GT-02287, the company’s lead drug candidate for the treatment of GBA1 Parkinson’s disease. Preclinical data demonstrated that GT-02287 can restore the function of the lysosomal enzyme glucocerebrosidase (GCase), which becomes misfolded and dysfunctional due to a GBA1 gene mutation, the most common genetic risk factor […]

Collaborators Boehringer Ingelheim and CDR-Life have commenced a Phase I evaluation of BI 771716, their antibody fragment-based treatment developed to preserve the vision of people with geographic atrophy (GA). Boehringer Ingelheim and CDR-Life entered a licensing agreement in May 2020 and announced the selection of an antibody fragment-based therapeutic candidate in September 2021. BI 771716 […]

Aulos Bioscience has revealed new safety and efficacy data from the Phase I dose escalation portion of its Phase I/II clinical trial of AU-007 in patients with unresectable locally advanced or metastatic solid tumour cancers. The data will be presented at the Society for Immunotherapy of Cancer (SITC) 38th Annual Meeting, being held in San […]